Passage Bio, Inc. Q2 FY2022 earnings call

Chip Cale highlighted progress in advancing clinical programs for CNS disorders, including strong momentum in the Imagine-1 trial for GM1 gangliosidosis with recruitment of Cohort 4, FDA clearance of IND for PBML04 in metachromatic leukodystrophy, ongoing recruitment in GALax-C trial for early infantile Krabbe disease, and expect to dose first patient in upliFT-D trial soon. Mark Forman reviewed clinical programs: PBGM01 for GM1 showed continued improvement in developmental milestones and biomarker data; GALax-C trial for Krabbe had first patient dosed with an adverse event of acute hydrocephalus but patient is stable; PBFT02 for frontotemporal dementia has identified patients and expects to dose soon; PBML04 for MLD received FDA IND clearance. Simona King noted strong cash position with ~$239.3M in cash at end of Q2, R&D expenses decreased to $26.8M from $33.1M, and G&A expenses decreased to $13M from $15.4M.

Expect to report initial safety and biomarker data from Cohorts 2 and 3 of the Imagine-1 trial in the second half of 2022. Anticipate reporting initial safety and biomarker data from a subset of GALax-C Cohort 1 by the end of 2022. Consistently, expect to dose the first patient in the upliFT-D trial shortly.

Forward-looking statements subject to risks and uncertainties causing actual results to differ. Safety event in first patient dosed with PBKR03 (acute hydrocephalus). Evaluation of financial situation and priorities impacting program focus and prioritization.

Q: On Krabbe and GM1 data release, are they expected in press release or medical meeting? On MLD clinical development decision?

A: Evaluating best way to release data, could be press release or medical meeting; MLD decision based on evaluation of financials and priorities.

Q: Update on PBKR03 safety events, how many additional patients recruited? On PBFT02 dosing?

A: First patient dosed with PBKR03 had acute hydrocephalus, patient stable; recruiting additional patients; PBFT02 expects to dose shortly due to patient identification efforts.

Q: FTD recruitment bottleneck, what's the issue?

A: Multifactorial, including patient identification, competition, eligibility; outreach efforts increasing genetic testing among FTD patients.

Q: CEO search update?

A: Board engaged Russell Reynolds for CEO search, active search with progress being made while executive team focuses on milestones and cash management.